Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with Hereditary Angioedema (HAE)
Hereditary angioedema (HAE) is a lifelong genetic condition that causes sudden, severe swelling in different parts of the body, such as the face, hands, gut, or throat. This clinical trial is testing an experimental treatment called NTLA-2002 in adults with HAE. NTLA-2002 uses gene-editing technology to turn down a specific protein in the blood that triggers swelling attacks. The main goals of the study are to check that the treatment is safe, see what side effects it might cause, and find out whether it reduces the number and severity of attacks people experience. The trial has two parts: Phase 1 checks different doses for safety, while Phase 2 tests how well the treatment works compared to a dummy drip (placebo). Taking part is voluntary, and participants will receive regular check-ups and close health monitoring throughout.
At a glance
What is this study about?
Hereditary angioedema (HAE) is a rare genetic condition where the body produces too much of a chemical that causes fluid to leak into tissues. This leads to unpredictable and often painful episodes of swelling in the skin, stomach, and sometimes the airway, which can be dangerous. Current treatments usually involve regular injections to prevent attacks or rescue medicines to treat them when they happen. This trial is investigating a new approach that aims to offer longer-lasting control.
The experimental medicine being tested is called NTLA-2002. It is an innovative gene-editing therapy designed to target and alter a specific gene in liver cells. By doing this, it aims to lower the levels of a protein called prekallikrein, which plays a central role in triggering swelling attacks. The goal is to stop swelling before it starts, potentially from a single treatment.
Because this is an early-stage study (Phase 1/2), the primary focus is participant safety. Doctors want to see how the body handles the medicine, monitor any side effects, and check drug levels in blood and urine samples. As the trial progresses, researchers will also track the number of swelling attacks participants experience to see if the treatment effectively reduces or stops them.
Key takeaways
- The study tests NTLA-2002, an experimental gene-editing therapy for adults with hereditary angioedema (HAE).
- It aims to treat the root cause of HAE by reducing the protein that triggers painful swelling attacks.
- The main goals are to check safety, monitor side effects, and measure reductions in monthly attacks.
- The treatment is given as an intravenous drip, followed by regular clinic check-ups and monitoring.
- Participation is voluntary, and you can withdraw at any point without affecting your routine care.
Who may be eligible?
This trial is open to adults aged 18 and over who have been diagnosed with hereditary angioedema (HAE). Both men and women can take part.
Because this is an early study using gene-editing technology, the trial team will look closely at your general health. You may need to have had a certain number of swelling attacks over recent months to help researchers judge whether the treatment works.
People with certain other serious medical conditions, severe liver or kidney problems, or those who are pregnant or planning a pregnancy may not be able to join. The study doctors will review your complete medical history and carry out screening tests to confirm if the trial is suitable for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have a confirmed medical diagnosis of hereditary angioedema (HAE).
- I still experience swelling attacks and keep track of when they occur.
- I am able and willing to attend regular hospital appointments and provide blood and urine samples.
- I am not currently pregnant or planning to become pregnant during the trial.
What does participation involve?
Taking part begins with a screening visit where doctors check your overall health, review your attack history, and perform blood and urine tests. If you are eligible, you will receive the study treatment through an intravenous drip (an infusion into a vein). In the Phase 2 part of the study, some participants will receive the active medicine and others will receive a simple salt-water drip (placebo), though those on placebo may be offered the active medicine later in the study.
After receiving the infusion, you will have regular clinic visits so the medical team can monitor your health, check your vital signs, and collect blood and urine samples. You will also be asked to keep an electronic diary or record to note down any swelling attacks you have, their severity, and whether you needed your normal rescue medication.
Because NTLA-2002 involves gene editing, the study includes a thorough follow-up period lasting several months to track long-term safety and measure how well the treatment works over time.
Potential risks and benefits
Locations (3)
- —UnverifiedGermany
- —UnverifiedFrance
- —UnverifiedNetherlands
Common questions
What is NTLA-2002?
NTLA-2002 is an investigational treatment that uses gene-editing technology (CRISPR) to permanently switch off a specific gene in the liver, reducing the protein that triggers swelling attacks.
Will I definitely receive the active medicine?
In Phase 1, everyone receives the active drug at varying doses. In Phase 2, some people will receive a harmless salt-water dummy drip (placebo) first, but there is an option to receive the real medicine later in the study.
Can I still take my emergency rescue medication if I have an attack?
Yes. Your safety comes first, and you will still be able to use your approved emergency rescue treatments if you experience a swelling attack during the study.
How is the treatment given?
NTLA-2002 is given as a single intravenous drip into a vein, usually done in a hospital clinic under close medical observation.
Do I have to pay to take part?
No, clinical trial medicines and study-related tests are provided free of charge, and travel expenses for hospital visits are usually reimbursed.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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